Interesting, thanks jrgodfre.
It's a recent update from the blog of Dr Roger Amos an UK hematologist in SCD.
About ACS , this was only a P1 and the authors in the article wrote: "Poloxamer 188 is safe to administer to patients with ACS, and preliminary data suggest that it may shorten its duration and the length of hospitalization in a dose related manner. Children appeared to benefit more than adults. "
ACS is an important secondary endpoint in EPIC, the difference could be that in EPIC patients in ACS aren't enrolled but they can be develop it when enrolled, a condition that could give some advantages to Vepoloxamer vs placebo due to early administration.IMO
The reasons of my position are mainly based only on the Interim statistical data of EPIC trial released by the company at the Investors & Analysts day on 7 October 2015. Its read is a duty for all investors, you can find it at Mast site,investors menu, SEC fillings.
The company reported a measured VOC duration of 79 hrs for all patients (250) in placebo and Vepoloxamer groups and they "supposed" a duration of 96 hrs in the placebo group.
Clearly my position is subject to the reliability of the data released by the company and on its suppositions. I count only on the faithfulness of the doctors that planned and supervised EPIC and on the professionalism of the biostatistical team of the Umass.
No biostock analysts, no pumpers/bashers, no company historians, no insitutionals, no gamblers on probabilities of biostock fail,no Thestreet ,....
If the Interim will reveal itself right I'll enjoy, but in all truth everything is behind my capabilities and possibilities.
for sure, it seems that arql07 has a very strange idea about how the trial is made. Very confused.
arql05 says: "They needed younger patients and needed parents willing to get a placebo for their child instead of the generic"
WHAT? Instead of the generic?
All the patients get SOC ! In addition they can get vepoloxamer or placebo.
arql07 again:"They have to remain there for 48 hrs, like the ones getting your drug, then go home and answer questions for 30 days."
All the patients are discharged when the VOC is finished, not after 48 hrs !!!
All the patients in the hospitals have a device that dose the painkillers on their request recording all operations and nobody knows if they are taking also Vepo or placebo.
30 days after discharge is the time of the follow up for a second VOC or for complications, in this case they are re-hospitalized.
All the patients already tested in case of another VOC can receive Vepo (not blinded EPIC-E trial) .
A great effort for a serious and huge P3 trial designed with FDA..
surely Mast will be an interesting target for a buyout. A bit high your target I'd be happy also with the half.
Clopidogrel, prasugrel already failed in SCD:
Ticagrelor is another platelet inhibitor via P2Y12 Receptor Antagonist , unlikely it will pass.
In any case it is not for VOC duratuion,
Maybe in combo therapy with Vepo could work or be synergic, then AZN should buyout MAST
Some points on the failed GALE cancer vaccine Neuvax
I start by sayng that my main interest in biotech is for the cancer immunotherapy , I followed for ages cancer vaccines trials and never I saw one of them that worked enough in P3 . Now the reasons are quite understand: the cancer is able to change and to turn off the attack of the immune system in few weeks or few months, then the result in survival usually is weak also if the early vaccine-response is strong. These therapies gave only few months of improvement, not enough for the statistical significance.
Furthermore, imo,the P2 results of Neuvax weren't so encouraging to pass to the Phase 3.
Luckily vaccines are knowing an amazing revival and very encouraging results when used in combo therapy with checkpoints inhibitor/activator of the Tcells that modulates the immune-system response in an opposite way that cancer does. Unluckily these trials are still in P1.
SCD is another story, the long experience with Poloxamer has permitted to know when it works better and has permitted to select some subgroups,at least 2 very large subgroups with good response.
We know that anticoagulants alone are insufficient to control the disease and the VOCs but acting on RBC structure like HU does and repairing also their damaged membranes with Vepo together with other effects,could give an adequate response.
Soon we'll see how good will be these numbers.
Shkreli is an ex-CEO incriminates in pharma scam,a bad person representing bad interestes, it's right to stop him immediately,saying clearly what he represent.
well done Culley!I appreciate CEO tweets
such a ridiculous post.
Poloxamer188 has been studied at least for 20 years by doctors, biochemists, university and hospital researchers, it has been tested on thousands of patients on several diseases with meticulous measurements but nobody noticed that its effect finishes after 6 hours.
Dr. Martin Emanuele took also part in the previous trial, he knows all details.
Only a compulsive basher like the Adam F. could find this kind of excuse to cheat some sucker investors.
Instead from tests come to light the importance to use Vepoloxamer early, it can change the VOC development repairing the bad red blood cells that cause the occlusion and reactivating the flow in the micro vessels.
Interesting in the results analysis will be to know how good is the early use of vepo in adults, that could fill the gap
with children in shorting VOC.
Just my opinion
yes, great analysis, but why do so many people heed a charlatan like Martin Shkreli?
He is only a skillful gambler in promoting his image but ultimately he remains an sophomoric young man , I doubt he is able to understand correctly scientific articles, he copies something here an there showing it as his expertise.
A waste of time for all and a risk for suckers.
Said that,the drugs trials are suited for every kind of speculations and uncertainties and then for any kind of play. This is a good reason to avoid messy windbags and base your choices on in-depth DDs.
Note that Martin S posted only the negative data of the Jama article forgetting the good data in children and in HU groups, exactly that EPIC is.
Incompetence or misinformation? Likely both. Luckily his tweets can't change a thing of the results and of the PPS after data readout. He is a desperate social climber.
EPIC is based on 2 very large "sub groups" or better is based on the reduction of a particular sub group: adults not taking HU.
Marty and Adam short all small biotechs because the P3 in small biotech often fail. No intelligence nor competence.
Martin S . furthermore for MSTX used only the bad side of previous P3 , typical dishonest reasoning to support his opinion.
for Vepo the change is insignificant.
Politically and economically for the world is not good for the West very bad. , maybe Chinese and Russian are happy to count always more.
For the Great Britain now the risk is to become the Little Britain . My hope is that Scotland and Ireland ask to join EU leaving Britain.